Benefits and Limitations of Real-World Patient-Reported Toxicity Symptom Monitoring for Guidelines and Care, as Perceived by Patients, Clinicians, and Guideline Developers

Abstract

Background: Toxicity monitoring should be modernized to include real-world patient-reported data. However, little is known about how stakeholders view the incorporation of real-world patient-reported toxicity symptoms into guidelines. This gap hinders the development of a sustained learning healthcare environment and limits the incorporation of this data into daily care.

Methods: This qualitative study, reported according to COREQ, involved interviews with 29 plus 10 chronic myeloid leukemia (CML) patients and 18 CML clinicians, including eight hematologists/guideline developers. The interviews were audio-recorded, transcribed, and independently coded in Atlas.ti. A framework, adapted from systematically sourced literature, was used for coding. Codes were assessed as either beneficial or limiting. An expert panel of all CML guideline developers completed and prioritized the identified knowledge gaps through a RAND-modified Delphi procedure.

Results: Thirty-one benefits and limitations of systematically monitoring patient-reported toxicity symptoms in the real world were identified. Compared to an existing framework, novel benefits centered around the use of aggregated data: Participants viewed real-world patient-reported toxicity symptoms as a way to systematically include patients' toxicity symptoms in the guidelines; personalize guideline advice; and fill knowledge gaps. The expert panel agreed on 14 knowledge gaps in chronic myeloid leukemia care that could be addressed through such data. Novel limitations focused on the suitability, acceptance, and applicability of toxicity symptom monitoring in routine clinical practice. Participants felt that this monitoring does not establish a causal link between medication and symptoms, and it has no added value over open conversation.

Conclusions: The benefits and limitations of adopting patient-reported real-world toxicity symptom monitoring need to be leveraged and addressed to ensure maximum value and uptake. Guideline developers viewed aggregated data as beneficial. The identified knowledge gaps provide concrete points of action for CML guideline development.

Keywords: chronic myeloid leukemia; evidence ecosystem; learning healthcare environment; patient‐reported outcome measures; real‐world evidence; toxicity monitoring.

FIGURE 1

Flowchart of the study.

FIGURE 2

The concept of the “Digital and Trustworthy Evidence Ecosystem” visualized with the production of evidence upstream and its impact downstream (reproduced with permission [72]). HER, Electronic health records; HTA, Health technology assessment.